At Children’s Hospital of Philadelphia, an 11-year-old girl with severe beta thalassamia, became the first pediatric patient to receive Zyntelgo, the first FDA-approved gene therapy for transfusion-dependent beta thalassamia.
Since receiving the therapy, the patient, Rahameen Nabeel, no longer requires blood transfusions and has seen dramatic improvements in her health and daily life, according to an April 28 news release from the hospital.
The inherited blood disorder affects hemoglobin production. Standard treatment would have required her to undergo transfusions every two to five weeks and regular monitoring for complications, like iron buildup.
In 2022, the FDA approved Zyntelgo, a gene therapy that targets the genetic cause of beta thalassamia by modifying the patients own stem cells to help with normal hemoglobin production.
